AuthorisedHuman Pharmacology (Phase I)- First administration to humansInterventional

A Phase 1 Study of AUTX-703 in Participants with Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes

This research trial is testing a new medication, AUTX-703, for adults living with certain blood cancers. Specifically, it's for people with acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS) that have come back after treatment or haven't improved with current therapies. This is a very early stage of research, known as a Phase 1 study. This means it's one of the first times this treatment is being given to people. The main aim is to find out if AUTX-703 is safe and what dose can be given without causing too many problems. It's an important step in developing new ways to fight these conditions.

At a glance

Status

Authorised

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

Auron Therapeutics Inc.

Enrolment target

Start

04 Feb 2026

Relapsed/refractory Acute Myeloid Leukemia or relapsed/refractory myelodysplastic syndromes

What is this study about?

This study is exploring a new medicine called AUTX-703. It's designed for adults who have been diagnosed with acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS). These are types of blood and bone marrow cancers. The study is specifically for those whose cancer has returned after previous treatments, or perhaps didn't respond well to treatments they've already received. Doctors sometimes call this 'relapsed' or 'refractory' cancer.

This trial is an early-stage study, known as 'Phase 1'. When a new medicine is developed, it goes through several stages of testing. Phase 1 is usually the very first time the medicine is given to people. The main purpose at this stage is not yet to see if it cures the disease, but to check if it's safe and to find the right dosage. Researchers want to understand how the body handles the new medicine and what side effects it might cause.

The findings from this study will help doctors decide if AUTX-703 should be tested further in larger groups of people. It's a crucial step in the long process of discovering new and better treatments for these challenging conditions, offering hope for patients in the future.

Key takeaways

This study is testing a new drug, AUTX-703, for certain blood cancers.
It's for adults with AML or MDS that has come back or hasn't responded to previous treatments.
This is an early-stage (Phase 1) study focused on safety and finding the right dose.
Participants will receive the new treatment and be closely monitored.
There are potential benefits and risks, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you must be an adult, aged 18 or older. Both men and women are welcome to take part.

You would be considered if you have acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS). Importantly, your condition must be either 'relapsed', meaning it has come back after treatment, or 'refractory', meaning it hasn't responded well to the treatments you've received so far. The study team will need to review your medical history in detail to make sure this trial is the right fit for your specific situation.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of Acute Myeloid Leukaemia (AML) or Myelodysplastic Syndromes (MDS)?
Has your AML or MDS relapsed (come back) or not responded to previous treatments?
Are you able to attend regular hospital/clinic appointments?
Are you prepared to receive an experimental new medication?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the hospital or clinic. During these visits, the study team would closely monitor your health. This typically includes physical examinations, blood tests, and sometimes other tests like bone marrow samples, to see how your body is responding to the treatment. You will receive the study medication, AUTX-703, as directed by the research team.

The research team will explain how long you would be expected to receive the treatment and how often you'd need to attend appointments. There will also be a follow-up period after you stop the treatment, to make sure you're doing well and to track any longer-term effects. The total time you spend in the study, from your first visit until the very last follow-up, would be fully explained to you before you decide to join.

Potential risks and benefits

While joining a study like this offers the potential to try a new treatment that might help your condition, it's important to remember that it's a very early-stage investigation. This means potential benefits are not guaranteed and might be unknown. There are also potential risks involved, as with any new medication. Possible side effects of AUTX-703 are not yet fully understood, and could include anything from mild discomfort to more serious reactions. The study doctors will carefully monitor you for any side effects and will explain all known or expected risks before you decide to participate. You are completely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is acute myeloid leukaemia (AML) and myelodysplastic syndromes (MDS)?

These are types of cancer that affect the blood and bone marrow, the spongy tissue inside your bones where blood cells are made.

What does 'relapsed' or 'refractory' mean?

It means your cancer has come back after treatment, or it hasn't responded well to the treatments you've had so far.

What is a Phase 1 study?

It's an early-stage study where a new medicine is given to people for the first time. The main goal is to check if it's safe and to find the right dose.

Will I get the new medicine?

Yes, everyone in this study receives the new medicine called AUTX-703, as this is a dose-finding and safety study. There's no placebo (dummy treatment) involved.

How long will I be in the study?

The study team will give you a clear idea of how long you might be receiving the treatment and how long the follow-up period will be, so you know the total duration before you agree to take part.